New FDA Approval: Deucravacitinib (Sotyktu), a First-In-Class Oral, Allosteric Tyrosine Kinase 2 Inhibitor

Relyvrio, developed by Amylyx Pharmaceuticals, is a combination treatment of sodium phenylbutyrate and taurursodiol, approved by the FDA in 2022 for ALS (“Lou Gehrig’s disease”). The approval was controversial, with an Advisory Committee initially voting against approval based on Ph. II data. Now, Ph. III data have confirmed the drug is no better than placebo in ALS, turning this phenotypic drug discovery story from two undergrads into a post-mortem highlighting the importance of clearly defined molecular mechanisms of action. This article reviews what happened and what we can learn.

This summary of September's major clinical updates features FDA approvals, drug withdrawals, anticipated regulatory decisions, notable trial results, new NDA submissions, clinical trial setbacks, and the initiation of new clinical trials.

This article highlights million-dollar molecules that were recently in the news with related patent applications that give clues to their structures and properties. This edition includes Maze’s glycogen synthase 1 (GYS1) inhibitors that were recently licensed to Sanofi, allosteric androgen receptor (AR) modulators that may be of interest to targeted protein degradation researchers, brain-penetrant HER2 and ROCK2 inhibitors, and MDM2 degraders.

Companies inked notable high-profile transactions in March 2024, including AbbVie's purchase of Landos Biopharma for $137.5M, AstraZeneca's $2.4B acquisition of Fusion Pharmaceuticals, and its subsequent deal to buy Amolyt for up to $1.05B. The FDA approved the first treatment for MASH. In parallel, expectations are high for the approval of Xcovery's ALK inhibitor, ensartinib. Praxis made headlines with PRAX-628, a next-generation oral NaV modulator, which showed anti-seizure efficacy in a Ph. IIa trial. Read below for more details on these and other drug discovery news stories from March 2024

We asked the global drug discovery community to nominate and vote on their favorite molecule from 2022, and the results are in. The 2022 winner, with the most overall votes across the ten finalist molecules , is BMS’ oral, deuterated allosteric TYK2 inhibitor, deucravacitinib, the first new treatment for plaque psoriasis in nearly a decade [...]